Clinical Trial 23346

• Overview

  Study Title:

  A Phase 3, Randomized, Double-blind, Add-on Study Evaluating the Safety and Efficacy of Navtemadlin Plus Ruxolitinib vs Placebo Plus Ruxolitinib in JAK Inhibitor-Naïve Patients with Myelofibrosis Who Have a Suboptimal Response to Ruxolitinib

  Summary:

  This clinical trial is evaluating whether addition of navtemadlin to ruxolitinib treatment will provide more clinical benefit than ruxolitinib alone for patients with Myelofibrosis who have a suboptimal response to ruxolitinib treatment alone.

  Objective:

  Primary Objectives: To compare SVR35 between Arm 1 and Arm 2. To compare TSS50 between Arm 1 and Arm 2. Secondary Objectives: To compare SVR25 between Arm 1 and Arm 2. To compare TSS30 between Arm 1 and Arm 2. To compare SVR35 between Arm 1 and Arm 2. To compare TSS50 between Arm 1 and Arm 2. To compare overall survival (OS) between Arm 1 and Arm 2. To compare progression-free survival (PFS) between Arm 1 and Arm 2. To compare duration of spleen response between Arm 1 and Arm 2 in suboptimal responders. To compare spleen response rate at any time between Arm 1 and Arm 2. To compare spleen size reduction as measured by palpation between Arm 1 and Arm 2. To compare the safety and tolerability of navtemadlin vs placebo as add-on therapy to ruxolitinib.

• Treatments

  Therapies:

  Therapy (NOS)

  Medications:

  Jakafi (Ruxolitinib); Navtemadlin (); Placebo (); Ruxolitinib ()

• Inclusion Criteria

  Inclusion Criteria for Ruxolitinib Alone Period:
  • Confirmed diagnosis of Primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF), or post-essential thrombocythemia myelofibrosis (post-ET MF), as assessed by the treating physician according to the World Health Organization (WHO) criteria
  • High, Intermediate-1, Intermediate-2 risk category International Prognosis System Score (IPSS)
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2
  • JAK-inhibitor treatment naive Inclusion Criteria for Randomized Period:
  • PMF, post-PV MF, or post-ET MF that is TP53WT as assessed by central testing
  • ECOG performance status of 0 to 2
  • Treatment with a stable dose of ruxolitinib
  • Suboptimal response to run-in ruxolitinib treatment

• Exclusion Criteria

  Exclusion Criteria for Ruxolitinib Alone Period:
  • Prior Splenectomy
  • Splenic irradiation within 3 months prior to the first dose
  • Prior B-cell lymphoma-extra large (BCL-XL), BET, MDM2, PI3K, PIM, or XPO1 inhibitors therapy or p53-directed therapy
  • Eligible for Bone Marrow Transplant
  • Peripheral blood or bone marrow blast count of 10 percent or greater Exclusion Criteria for Randomized Period:
  • Elevated white blood cell count that doubles (or more) during ruxolitinib treatment
  • Peripheral blood or bone marrow blast count of 10 percent or greater

If you are interested in learning more about clinical trials, our clinical trial navigators can discuss your options and recommend opportunities that may be suitable for you. Call 813-745-6100 or 1-800-679-0775 (toll-free) or submit a clinical trials inquiry form.